Key Takeaways RARE and MREO fell after delaying new data from the phase III UX143 Orbit study to year-end. The Orbit study continues as planned, with final data expected by the end of 2025. Interim safety review found UX143 acceptable, allowing studies to proceed without changes.Ultragenyx Pharmaceutical (RARE) and partner, Mereo BioPharma (MREO) , have reported an update on the progress of the mid-to-late-stage Orbit study, which is evaluating UX143 (setrusumab) for the treatment of osteogenesis imperfec ...

Ultragenyx Pharmaceutical Inc. RARE and Mereo BioPharma Group plc MREO stocks are plunging on Thursday.What Happened?The companies on Wednesday announced that the randomized, placebo-controlled Phase 3 portion of the Orbit study evaluating UX143 (setrusumab) in pediatric and young adult patients with osteogenesis imperfecta (OI) is progressing toward a final analysis around the end of the year, consistent with the original plan.OI, also known as brittle bone disease, is a group of genetic disorders that mak ...

Key Takeaways The FDA granted Breakthrough Therapy designation to Ultragenyx's GTX-102 for Angelman syndrome. GTX-102 showed rapid, lasting improvements in AS patients during a phase I/II study over up to 3 years. RARE is enrolling for a phase III Aspire study and planning a broader phase II/III Aurora study for GTX-102.Ultragenyx Pharmaceuticals (RARE) announced that the FDA has granted the Breakthrough Therapy designation to its investigational antisense oligonucleotide therapy, GTX-102 (apazunersen), t ...

Corporate Presentation May 2025 Confidential and Proprietary Forward looking statements Cautionary note regarding forward-looking statements: This presentation contains forward-looking statements, including, but not limited to, statements regarding our expectations, estimates, assumptions, and projections regarding our future operating results and financial performance, including our expectations for profitability in 2027, anticipated cost or expense management, plans with respect to commercializing our pro ...